Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

  • End date
    Dec 20, 2023
  • participants needed
  • sponsor
    Memorial Sloan Kettering Cancer Center
Updated on 8 April 2021


This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.

Condition childhood ALL, Pancytopenia, Blood disorder, Preleukemia, Hemophagocytic lymphohistiocytosis, Acute myeloid leukemia, miller-dieker syndrome, Hemoglobinopathy, MYELOPROLIFERATIVE DISORDER, MYELODYSPLASTIC SYNDROME, Myelodysplastic Syndromes (MDS), Acute Myelogenous Leukemia (AML), Inherited Metabolic Disorders, Myeloproliferative Neoplasms, Lymphocytic Leukemia, Acute, Hematological Disorders, MDS, leukemia, acute lymphoblastic, myelodysplasia, myelodysplastic syndromes, myeloproliferative neoplasm, myeloproliferative disorders, hemoglobinopathies, bone marrow failure, myelodysplastic syndrome (mds), acute lymphoid leukaemia, acute lymphocytic leukemia, acute lymphoblastic leukemia (all), acute myelogenous leukemia, anll, acute myeloblastic leukemia, Therapy-Related AML and MDS, Therapy-Related Acute Myeloid Leukemia, T-AML, Therapy-Related AML and MDS, Therapy-Related Acute Myeloid Leukemia, T-AML, MPD Withou Myelofibrosis, NHL or HL
Treatment mycophenolate mofetil, busulfan, Fludarabine, Clofarabine, Cyclosporine-A, Cord Blood Graft
Clinical Study IdentifierNCT04644016
SponsorMemorial Sloan Kettering Cancer Center
Last Modified on8 April 2021


Yes No Not Sure

Inclusion Criteria

Is your age less than or equal to 21 yrs?
Gender: Male or Female
Do you have any of these conditions: myelodysplastic syndromes or MYELOPROLIFERATIVE DISORDER or MDS or Myeloproliferative Neoplasms or anll or MYELODYSPLASTIC SYNDROME or T-AML or Acute ...?
Do you have any of these conditions: miller-dieker syndrome or Myelodysplastic Syndromes (MDS) or acute myeloblastic leukemia or anll or MYELODYSPLASTIC SYNDROME or myelodysplastic syndro...?
Do you have any of these conditions: acute lymphocytic leukemia or anll or Myelodysplastic Syndromes (MDS) or Blood disorder or MPD Withou Myelofibrosis or myelodysplasia or Acute Myeloge...?
Do you have any of these conditions: Hemophagocytic lymphohistiocytosis or hemoglobinopathies or leukemia, acute lymphoblastic or Therapy-Related AML and MDS or T-AML or Myelodysplastic S...?
Do you have any of these conditions: Myeloproliferative Neoplasms or Hemophagocytic lymphohistiocytosis or acute myelogenous leukemia or NHL or HL or MPD Withou Myelofibrosis or Therapy-R...?
Do you have any of these conditions: Myeloproliferative Neoplasms or acute lymphocytic leukemia or NHL or HL or hemoglobinopathies or acute myelogenous leukemia or myelodysplastic syndrom...?
Do you have any of these conditions: acute myeloblastic leukemia or Acute Myelogenous Leukemia (AML) or Blood disorder or acute lymphoid leukaemia or hemoglobinopathies or MDS or Myelopro...?
Age and Donor Status
Patients with age 21 years at time of consent with no available and suitably
matched related or unrelated donor in the required time period
I. Acute myelogenous leukemia (AML)
Complete first remission (CR1) at high risk for relapse such as any of the following
Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS)
Therapy-related AML (t-AML)
White cell count at presentation > 100,000
Presence of extramedullary leukemia at diagnosis
Any unfavorable subtype by FAB or WHO classification
High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities
Requirement for 2 or more inductions to achieve CR1
Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction
Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy
Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician
Other high-risk features not defined above
Complete second remission (CR2)
Primary refractory or relapsed AML with less than 10% blasts by bone marrow morphology. Patients with cytogenetic, flow cytometric, or molecular abnormalities in 10% of cells are eligible
II. Acute lymphoblastic leukemia (ALL)
Complete first remission (CR1) at high risk for relapse such as any of the following
White cell count at presentation > 30,000 for B-cell lineage and > 100,000 for T-cell lineage
Presence of any high-risk cytogenetic abnormalities such as t (9;22), t (1;19), t (4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality
Failure to achieve complete remission (CR) after four weeks of induction therapy
Persistence or recurrence of MRD on therapy
Any patient unable to tolerate consolidation and/or maintenance chemotherapy as would have been deemed appropriate by the treating physician
Other high-risk features not defined above
Complete second remission (CR2)
Primary refractory or relapsed ALL with MRD disease after antibody therapy (e.g., blinatumomab, inotuzumab, other) and/or CAR-T cell therapy
III. Other acute leukemias
Leukemias of ambiguous lineage or of other types (e.g. blastic plasmacytoid
dendritic cell neoplasm) with less than 5% blasts by BM morphology. Patients
with persistent/relapsed disease with cytogenetic, flow cytometric or
molecular aberrations in 5% of cells are eligible
IV. Myelodysplastic Syndrome (MDS) / Myeloproliferative Disorders (MPD) other
International prognostic scoring system (IPSS) risk score of INT-2 or high risk at the time of diagnosis
Any IPSS risk category if life-threatening cytopenia(s) exists
Any IPSS risk category with karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia
MDS/ myeloproliferative disorder overlap syndromes without myelofibrosis
MDS/ MPD patients must have less than 10% bone marrow myeloblasts and ANC 0.2 (growth factor supported if necessary) at transplant work-up. V. Non-Hodgkin lymphoma (NHL) or Hodgkin lymphoma (HL) at high-risk of relapse or progression if not in remission
Eligible patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histology) in CR
Eligible patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with stable disease/ CR/ PR with no single lesion equal to or more than 5 cm
Eligible patients with HL will be those without progression of disease (POD) after salvage chemotherapy with no single lesion 5 cm
VI. Inherited Metabolic Disorders [also see EBMT Handbook for discussion on
patient eligibility for allogeneic transplant; in general, patients are
considered early in the disease course, before they develop neurologic
symptoms (46)]
Hurler Syndrome
Hunter (MPS 2 - early disease)
Sly syndrome (MPSVIII)
Metachromatic Leukodystrophy
Globoid (GLD)
VII. Non-Malignant disorders (other) [also see EBMT Handbook for criteria for
transplant (46)]
Bone Marrow Failure syndromes
Immunodeficiencies, including HLH
Organ Function and Performance Status Criteria
Karnofsky or Lansky score 70% (see Appendix)
Bilirubin 1.5 mg/dL (unless benign congenital hyperbilirubinemia)
ALT 3 x upper limit of normal
Pulmonary function (spirometry and corrected DLCO) 50% predicted (corrected for hemoglobin)
Left ventricular ejection fraction 50%
Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) less than or equal to 7
Renal: serum creatinine 1.5x normal for age. If serum creatinine is outside the normal range, then CrCl > 50 mL/min/1.73m2 (calculated or estimated) or GFR (mL/min/1.72m2) >30% of predicted normal for age
Normal GFR in Children and Young Adults (Age) : Mean GFR +- SD (mL/min/1.73
week: 40.6 + / - 14.8 2-8 weeks: 65.8 + / - 24.8 >8 weeks: 95.7 + / - 21.7
-12 years: 133.0 + / - 27.0 13-21 years (males): 140.0 + / - 30.0 13-21 years
(females: 126.0 + / - 22.0
GFR, glomerular filtration rate; SD, standard deviation greater than 2 years
old: Normal GFR is 100 mL/ min. Infants: GFR must be corrected for body surf
ace area
For metabolic diseases: disease status to be evaluated according to EBMT
Handbook [45]
Graft Criteria
CB units will be selected according to the current MSKCC unit selection
algorithm. High resolution 8 allele HLA typing and recipient HLA antibody
profile will be performed. Cord unit selection will occur based on HLA-match
total nucleated cell (TNC) and CD34+ cell dose adjusted per patient body
weight. The cord bank of origin will also be considered. Donor specific HLA
antibodies, if present, will also be taken into consideration and may
influence the selection of the graft. CB graft will consist of one or two CB
units (CBU) based on MSKCC selection algorithm
Each CB unit must be at least 3/8 HLA-matched to the patient considering high-resolution 8-allele HLA typing
For malignant diseases follow MSKCC CBU selection algorithm
For non-malignant diseases, CBU will be required to have > 5 x 107 TNC/kg; high HLA allele level match is preferable

Exclusion Criteria

Inadequate performance status/ organ function
Advanced metabolic disease (EBMT handbook)
Active CNS leukemic involvement
Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy
Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis
Autologous stem cell transplant within the preceding 6 months
Any prior allogeneic stem cell transplant
Active and uncontrolled infection (bacterial/fungal/viral) at time of transplantation
HIV infection
Seropositivity for HTLV-1
Pregnancy or breast feeding
Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests
Clear my responses

How to participate?

Step 1 Connect with a site
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact


Phone Email

Please verify that you are not a bot.

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider


Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 



Reply by • Private

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note